Timeline

The journey from research to potential treatment.

How development progresses

The development of gene therapy follows a structured path, moving from early research to clinical application. Each stage builds on the previous one and must meet strict scientific and regulatory standards.

GAND GENE THERAPY PROGRAM

DEVELOPMENT ROADMAP

Development timeline from research and preclinical validation toward regulatory preparation.

LAST UPDATED JUNE 2026
Development activity
Planning phase
Regulatory milestone
ACTIVITY / WORKSTREAM
2025
2026
2027
2028
DEVELOPMENT PERIOD
JAN FEB MAR APR MAY JUN JUL AUG SEP OCT NOV DEC
JAN FEB MAR APR MAY JUN JUL AUG SEP OCT NOV DEC
JAN FEB MAR APR MAY JUN JUL AUG SEP OCT NOV DEC
JAN FEB MAR APR MAY JUN JUL AUG SEP OCT NOV DEC
01
Construct development
02
iPS development
03
In vitro testing HEK293T and SH-SY5Y cells
04
Brain organoid testing
05
GAND mouse development
Planning
Generation + study
06
Mice efficacy study
Protocol planning
Study
07
Manufacturing cells + 2L
08
Manufacturing (50L)
09
Regulatory enabling study GLP mice
Protocol planning
Study
10
Regulatory documentation preparation
11
CTIS / IND Regulatory milestone
CTIS / IND
12
Child dosed

The process begins with fundamental research and understanding of the gene and disease mechanism. This is followed by preclinical development, where potential therapies are tested in controlled environments.

If successful, the therapy moves into clinical trials, where safety and effectiveness are evaluated in patients. Only after passing these stages can a treatment become widely available.

Key development phases

Girl with GATAD2B syndrome (GAND)

A long but meaningful journey

Developing a gene therapy can take several years. While the timeline may seem long, each step is essential to ensure safety, effectiveness, and long-term impact.

Every milestone reached represents real progress toward a future treatment.